Go to the profile of Winkler G. Weinberg, MD
Winkler G. Weinberg, MD
Dr. Wink Weinberg has been an certified Infectious Disease specialist since 1984. He is author of No Germs Allowed! (How to avoid infectious diseases).

The Father of Modern Medicine — May Not Be a Father!

Gertrude Elion

Thank goodness for Big Pharma! And the one company we have the most to thank is GlaxoSmithKline (GSK). GSK has led the way in inventing Modern Medicine, and they are about to launch us to the next level of sophistication in health, both on the scientific forefront and in the realm of economics.

Modern medicine really began during and shortly after World War II. During the war we saw many new developments, most prominently intravenous fluid resuscitation and penicillin. These were lunges forward into the modern era, but no one could even imagine then what was to come next.

The second half of the 20st Century saw the birth of cancer chemotherapy, organ transplantation, and antiviral therapy. It is a little known fact that these came about thanks to GSK innovations. The 21st Century is bringing us genetic approaches, including pharmacogenomics and gene therapy. Once again GSK is again paving the way.

Gertrude Belle Elion and shared the 1988 Nobel Prize in Physiology or Medicine with George H. Hitchings and Sir James Black. Dr. Elion was Head of the Department of Experimental Therapy for Burroughs Wellcome, a company that later morphed into GSK by mergers and acquisitions. Dr. Hitchings also worked for the Wellcome Research Laboratories. [Dr. Black was awarded the prize for a different line of research.] Elion’s and Hitchings’ inventions include:

Dr. Elion officially retired in 1983. Despite her retirement, she continued working almost full-time at the GSK labs, and participated in the adaptation of azidothymidine (AZT), which, approved in 1987, became the first drug useful for treatment of HIV/AIDS.

This century we have seen the nascence of gene based medicine. I am proud to say that I was one of many researchers involved in studies showing that a human genetic marker (HLA-B*5701) could predict that person’s reaction to a drug (abacavir). This was published in 2008 and was the first clinical application of pharmacogenomics. GSK conducted the research. This test is still part of the daily practice of medicine.

Next, GSK created the first injectable, long acting HIV medicine. One shot can last as long as 3 months. At first consideration, this may seem like more of a tweak than a major advance, but if you think about it for prevention rather than treatment, you will see that it can change the world. Treating HIV-positive individuals, and giving pre-exposure prophylaxis to HIV-negative individuals who are at risk, may finally decrease the incidence of HIV infection. In fact, this approach theoretically has the potential — after decades — to rid mankind of HIV. Take Australia, for example: Figures suggest there are now less than 100 AIDS cases per year in Australia, and they are nearly all caught and reversed by bringing the underlying HIV infection under control. The Australian Federation of AIDS Organisations have said that AIDS no longer constitutes a public health emergency there.

If we are going to use drugs to control HIV, as no vaccine is in site, we need an advance on the prevention side. Healthy individuals often fail to adhere with taking a pill every day for a disease they don’t have. But if the long-acting, injectable GSK1265744 proves to be effective, it might just be convenient enough to make pre-exposure HIV prophylaxis widely accepted.

Now on to gene therapy. There have been some successes with gene therapy for local diseases, as for rare eye diseases, but it appears that the first clinically relevant gene therapy for a systemic disease will be for X-linked severe combined immunodeficiency (SCID), commonly known as “bubble boy disease.” GSK recently won approval in Europe for a drug named Strimvelis that will first be sold in Itlaly. It is a stem cell gene therapy that can replace these baby’s missing adenosine deaminase (ADA), a protein which is required for the production of lymphocytes.

In a first on the Pharmaco-Economic front, GSK has agreed to refund the cost of Strimvelis to Italy for cases in which it does not work. The price tag on Strimvelis is $665,000. This is the list price for a one-time treatment, a record-breaker. But it is only given once, and without it SCID is fatal, usually in the first year of life. In the initial trials, only 18 children were treated (this is an extremely rare disease); 15 were cured. The disease is so rare that it will not “move the needle” of GSK revenue, and it will not “break the budget” for health plans. However, does this mean that we are we broaching the new era of gene therapy? Is there a new pay-for-performance reimbursement model that will be duplicated in the future? Are we witnessing a tectonic shift in the healthcare industry?

And now into the future! GlaxoSmithKline has also been quietly pioneering an entirely new scientific field: bioelectronic medicines. The basic idea is to build miniaturized implantable devices that could precisely measure nerve impulses throughout the body, and that could subsequently correct any irregular impulses associated with diseases such as arthritis, asthma, hypertension, and diabetes.

Recently, GSK took a major step toward making this moonshot a reality by signing a joint venture agreement with Alphabet’s Verily Life Sciences to create the first generation of bioelectronic medicines for chronic diseases. The new company will be called Galvani Bioelectronics, with Glaxo claiming 55%, and Verily 45%, of the new entity.

I am grateful that I live in a time with fantastic medical advances. I am even more grateful that my children and their children will benefit from Modern Medicine more than I can even imagine.

Winkler G. Weinberg, MD

I have been an Infectious Disease physician for over 3 decades. I am the author of No Germs Allowed!. If you liked this article, check out the book on Amazon.

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  • Go to the profile of Winkler G. Weinberg, MD

    Winkler G. Weinberg, MD

    Dr. Wink Weinberg has been an certified Infectious Disease specialist since 1984. He is author of No Germs Allowed! (How to avoid infectious diseases).